CA2909868C - Composes et procedes pour absorption cellulaire amelioree - Google Patents

Composes et procedes pour absorption cellulaire amelioree Download PDF

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CA2909868C
CA2909868C CA2909868A CA2909868A CA2909868C CA 2909868 C CA2909868 C CA 2909868C CA 2909868 A CA2909868 A CA 2909868A CA 2909868 A CA2909868 A CA 2909868A CA 2909868 C CA2909868 C CA 2909868C
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certain embodiments
compound
modified oligonucleotide
modified
nucleoside
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CA2909868A1 (fr
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Balkrishen Bhat
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Regulus Therapeutics Inc
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Regulus Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/999—Small molecules not provided for elsewhere

Abstract

L'invention concerne des oligonucléotides modifiés conjugués qui sont complémentaires d'un ARN cible. Le conjugué facilite l'absorption cellulaire de l'oligonucléotide modifié, ce qui permet d'obtenir une puissance améliorée.
CA2909868A 2013-05-01 2014-04-30 Composes et procedes pour absorption cellulaire amelioree Active CA2909868C (fr)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201361818441P 2013-05-01 2013-05-01
US201361818447P 2013-05-01 2013-05-01
US61/818,441 2013-05-01
US61/818,447 2013-05-01
US201361822115P 2013-05-10 2013-05-10
US61/822,115 2013-05-10
US201361895708P 2013-10-25 2013-10-25
US61/895,708 2013-10-25
PCT/US2014/036136 WO2014179445A1 (fr) 2013-05-01 2014-04-30 Composés et procédés pour absorption cellulaire améliorée

Publications (2)

Publication Number Publication Date
CA2909868A1 CA2909868A1 (fr) 2014-11-06
CA2909868C true CA2909868C (fr) 2021-10-19

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
CA2909868A Active CA2909868C (fr) 2013-05-01 2014-04-30 Composes et procedes pour absorption cellulaire amelioree

Country Status (11)

Country Link
US (3) US9506030B2 (fr)
EP (1) EP2992096B1 (fr)
JP (1) JP6649248B2 (fr)
CN (1) CN105164261B (fr)
AU (1) AU2014259953B2 (fr)
CA (1) CA2909868C (fr)
HK (1) HK1221257A1 (fr)
IL (1) IL241973B (fr)
NZ (1) NZ630921A (fr)
TW (1) TWI680767B (fr)
WO (1) WO2014179445A1 (fr)

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US9506030B2 (en) 2016-11-29
CA2909868A1 (fr) 2014-11-06
JP2016518841A (ja) 2016-06-30
AU2014259953B2 (en) 2020-07-02
CN105164261A (zh) 2015-12-16
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AU2014259953A1 (en) 2015-11-05
HK1221257A1 (zh) 2017-05-26
US10941400B2 (en) 2021-03-09
CN105164261B (zh) 2022-03-18
NZ630921A (en) 2017-12-22
EP2992096A1 (fr) 2016-03-09
TWI680767B (zh) 2020-01-01
US20170096668A1 (en) 2017-04-06
US10240151B2 (en) 2019-03-26
WO2014179445A1 (fr) 2014-11-06
US20150031130A1 (en) 2015-01-29
IL241973B (en) 2019-11-28
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US20190309292A1 (en) 2019-10-10

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